BioMarin Pharmaceutical
The purpose of this study is to evaluate i) the effect of multiple doses of vosoritide and ii) the effect of the therapeutic dose of vosoritide compared to human growth hormone (hGH)(hGH; only in the United States), in children with idiopathic short stature (ISS).
Idiopathic Short Stature
Vosoritide Injection
Human Growth Hormone
Placebo
PHASE2
Following a minimum 6 month observational period in which baseline growth is assessed, participants in the vosoritide and placebo groups will complete a minimum of 6 months of randomized treatment (maximum of 6 months of placebo treatment), followed by open-label treatment with vosoritide until they reach near-final adult height, or at least 16 years of age for females or 18 years of age for males, whichever comes later. Participants randomized to the hGH group will receive open-label hGH for a minimum of 4 years.
| Study Type : | INTERVENTIONAL |
| Estimated Enrollment : | 100 participants |
| Masking : | QUADRUPLE |
| Primary Purpose : | TREATMENT |
| Official Title : | A Phase 2, Randomized, Controlled, Multicenter Study of Vosoritide in Children With Idiopathic Short Stature |
| Actual Study Start Date : | 2024-10-21 |
| Estimated Primary Completion Date : | 2026-06 |
| Estimated Study Completion Date : | 2036-12 |
Information not available for Arms and Intervention/treatment
| Ages Eligible for Study: | 3 Years to 11 Years |
| Sexes Eligible for Study: | ALL |
| Accepts Healthy Volunteers: |
Want to participate in this study, select a site at your convenience, send yourself email to get contact details and prescreening steps.
RECRUITING
Consano Clinical Research, LLC
California City, California, United States, 78231
RECRUITING
Rady Children's Hospital - San Diego
California City, California, United States, 92123
RECRUITING
Children's Hospital of Orange County Main Campus - Orange
California City, California, United States, 92868
RECRUITING
Children's Hospital Los Angeles
Los Angeles, California, United States, 90027
RECRUITING
Children's Hospital Oakland Research Institute
Oakland, California, United States, 94609
RECRUITING
Center Of Excellence in Diabetes and Endocrinology
Sacramento, California, United States, 95821
RECRUITING
Lundquist Institute for Biomedical Innovation (LA BioMed)
Torrance, California, United States, 90502
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The Children's National Research Institute
Washington D.C., District of Columbia, United States, 20012
RECRUITING
Nemours Children's Specialty Care Jacksonville
Jacksonville, florida, United States, 32207
RECRUITING
Nicklaus Children's Hospital
Miami, florida, United States, 33155
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Optimal Research Sites
Orange City, florida, United States, 32763
RECRUITING
Nemours Children's Health System - Corporate Headquarters
Pensacola, florida, United States, 32514
RECRUITING
Centricity Research
Columbus, Georgia, United States, 31904
RECRUITING
St. Luke's Children's Endocrinology
Boise, Idaho, United States, 83712
RECRUITING
Rocky Mountain Clinical Research - Idaho Falls
Idaho Falls, Idaho, United States, 83404
RECRUITING
University of Iowa Stead Family Children's Hospital
Iowa City, Iowa, United States, 52242
RECRUITING
Kentucky Children's Hospital
Lexington, Kentucky, United States, 40536
RECRUITING
University of Louisville School of Medicine
Louisville, Kentucky, United States, 40202
RECRUITING
Velocity Clinical Research - MedPharmics - Lafayette
Lafayette, Louisiana, United States, 70508
WITHDRAWN
Ochsner Health Center for Children - New Orleans
New Orleans, Louisiana, United States, 70121
RECRUITING
University of Michigan
Ann Arbor, road cancer, United States, 48109
RECRUITING
University of Minnesota Masonic Children's Hospital
Minneapolis, Minnesota, United States, 55454
RECRUITING
UBMD Pediatrics
Buffalo, New York, United States, 14203
WITHDRAWN
NYU Langone Ambulatory Care Garden City
Garden City, New York, United States, 11530
RECRUITING
Atlantic Health Children's Specialty Center at Morristown
New York, New York, United States, 07960
RECRUITING
The Clinical and Translational Science Institute
New York, New York, United States, 10016
RECRUITING
Icahn School of Medicine at Mount Sinai
New York, New York, United States, 10029
RECRUITING
Children's Hospital at Montefiore
The Bronx, New York, United States, 10467
RECRUITING
University of North Carolina Children's Specialty Clinic
Chapel Hill, North Carolina, United States, 27514
RECRUITING
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
RECRUITING
Prisma Health Children's Hospital - Midlands
Columbia, South Carolina, United States, 29203
RECRUITING
University of Texas Southwestern Medical Center
dallas, Texas, United States, 75390
RECRUITING
Cook Children's Endocrinology
Fort Worth, Texas, United States, 76104
RECRUITING
Baylor College of Medicine
Houston, Texas, United States, 77030
RECRUITING
Seattle Children's Hospital
Seattle, Washington, United States, 98105
RECRUITING
Murdoch Children's Research Institute
Parkville, Victoria, Australia, 3052
RECRUITING
Angers University Hospital Center
Angers, France, 49 933
RECRUITING
Timone Hospital - Children
Marseille, France, 13005
RECRUITING
Toulouse University Hospital - Children's Hospital
Toulouse, France, 31059
RECRUITING
Bicetre Hospital
The Kremlin-Bicêtre, Île-de-France Region, France, 94270
RECRUITING
Saarland University Hospital
Homburg, Saarland, Germany, 66421
RECRUITING
University Hospital - Meyer Pediatric Hospital
Florence, Florence, Italy, 50139
RECRUITING
Bambino Gesù Pediatric Hospital
Roma, Rome, Italy, 00165
RECRUITING
IRCCS Giannina Gaslini Institute
Genova, Italy, 16147
RECRUITING
Seoul National University Bundang Hospital
Seongnam-si, Gyeonggi-do, South Korea, 13620
RECRUITING
Inha University Hospital
Incheon, South Korea, 22332
RECRUITING
Chungnam National University Sejong Hospital (CNUSH)
Sejong, South Korea, 30099